Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical
company developing novel therapies for the management of central
nervous system (CNS) disorders, today announced that the
development plan for AXS-12 for the treatment of narcolepsy has
been expedited following a Breakthrough Therapy meeting with the
U.S. Food and Drug Administration (FDA). AXS-12 is a novel, oral,
highly selective and potent norepinephrine reuptake inhibitor for
the treatment of narcolepsy.
The expedited development plan includes one
Phase 3 efficacy trial, which, along with the previously completed
Phase 2 CONCERT trial, will be used to support the filing of an NDA
(New Drug Application) for approval of AXS-12 for the treatment of
cataplexy in narcolepsy. The planned Phase 3 trial will be a
randomized, double-blind, placebo-controlled, parallel-group study.
Axsome intends to initiate this trial in 1Q 2021. Patients
completing this trial will be eligible to enroll in an open-label
safety extension study.
Also, as part of the expedited development plan,
it was established that the data from numerous short-term and
long-term clinical trials of patients treated with reboxetine, and
the data from a full range of completed nonclinical studies with
reboxetine, previously obtained through an exclusive license from
Pfizer, can be used to support the filing of an NDA for AXS-12.
“Axsome is very pleased with the FDA feedback
from our recent Breakthrough Therapy meeting, which resulted in an
expedited path to a potential NDA filing for AXS-12 in the
treatment of cataplexy in narcolepsy,” said Herriot Tabuteau, MD,
Chief Executive Officer of Axsome. “Narcolepsy is a debilitating
condition that interferes with almost every aspect of a patient’s
life. Our approach to the development of AXS-12 for narcolepsy
reflects our commitment to accelerating the innovation of
potentially life-changing medicines for the many patients living
with serious CNS disorders.”
Breakthrough Therapy Meeting
Outcomes
- One Phase 3
trial of AXS-12 in patients with narcolepsy will be conducted to
support, along with the completed Phase 2 CONCERT trial, an NDA
filing for AXS-12 in the treatment of cataplexy in narcolepsy.
- The existing
short-term and long-term safety database of more than 2,500
patients treated with reboxetine, previously obtained through an
exclusive license from Pfizer, along with the safety data from the
completed and planned studies of AXS-12 in patients with
narcolepsy, would be sufficient to support an NDA filing for
AXS-12.
- Certain
existing, completed clinical pharmacology (e.g. human
pharmacokinetic) studies of reboxetine, previously obtained through
an exclusive license from Pfizer, are considered sufficient to
support an NDA filing for AXS-12.
- The extensive
package of completed nonclinical studies of reboxetine, previously
obtained through an exclusive license from Pfizer, are considered
sufficient to support an NDA filing for AXS-12.
In August 2020, Axsome received Breakthrough
Therapy designation from the FDA for AXS-12 for the treatment of
cataplexy in narcolepsy. A Breakthrough Therapy designation is
granted to potentially expedite development and review timelines
for a promising investigational medicine when preliminary clinical
evidence indicates it may demonstrate substantial improvement on
one or more clinically significant endpoints over available
therapies for a serious or life-threatening condition. The
Breakthrough Therapy designation for AXS-12 for the treatment of
cataplexy in narcolepsy was supported by the positive results from
the Phase 2 CONCERT study, a randomized, double-blind,
placebo-controlled, crossover, multicenter U.S. trial. In the
trial, 21 patients with a diagnosis of narcolepsy with cataplexy
were treated for 2 weeks with AXS-12 or with placebo, followed by a
crossover to the other treatment after a 1-week down-titration and
washout period. AXS-12 met the primary endpoint demonstrating a
highly statistically significant reduction from baseline in the
mean weekly number of cataplexy attacks, averaged for the 2-week
treatment period (overall treatment effect), as compared to placebo
(p<0.001), and at the end of the 2-week treatment period
(p=0.002). AXS-12 also significantly improved excessive daytime
sleepiness compared to placebo, as measured by the Epworth
Sleepiness Scale and by the frequency of inadvertent naps (p=0.003
and p<0.001, respectively). In addition, AXS-12 significantly
improved cognitive function compared to placebo over the 2-week
treatment period as measured by the Ability to Concentrate item of
the Narcolepsy Symptom Assessment Questionnaire (p<0.001).
AXS-12 was well tolerated in this trial with the most commonly
reported adverse events being anxiety, constipation, and
insomnia.
Conference Call Information
Axsome will host a conference call and webcast
with slides today at 8:00 AM Eastern to discuss the expedited
development plan for AXS-12 for the treatment of narcolepsy
following a Breakthrough Therapy meeting with the FDA. To
participate in the live conference call, please dial (844) 698-4029
(toll-free domestic) or (647) 253-8660 (international), and use the
passcode 9584742. The live webcast can be accessed on the “Webcasts
& Presentations” page of the “Investors” section of the
Company’s website at axsome.com. A replay of the webcast will be
available for approximately 30 days following the live event.
About FDA Breakthrough Therapy
Designation
Breakthrough Therapy designation is granted by
the FDA in order to expedite the development and review of drugs
for serious or life-threatening conditions. In order to receive
Breakthrough Therapy designation, a drug must demonstrate
preliminary clinical evidence that the drug may have substantial
improvement on at least one clinically significant endpoint over
available therapy. Breakthrough Therapy designation provides an
organizational commitment involving senior managers from the FDA,
more intensive FDA guidance on an efficient drug development
program, and greater access to and more frequent communication with
the FDA throughout the entire drug development and review process.
It also provides the opportunity to submit sections of a New Drug
Application (NDA) on a rolling basis, where the FDA may review
portions of the NDA as they are received instead of waiting for the
entire NDA submission. In addition, Breakthrough Therapy designated
products are eligible for Priority Review, where the FDA has a goal
to take action on an application within six months, as opposed to
ten months under standard review. Breakthrough Therapy designation
does not change the standards for approval.
About
Narcolepsy
Narcolepsy is a serious and debilitating
neurological condition that causes dysregulation of the sleep-wake
cycle and is characterized clinically by excessive daytime
sleepiness, cataplexy, hypnagogic hallucinations, sleep paralysis,
and disrupted nocturnal sleep. Narcolepsy afflicts an estimated
185,000 individuals in the U.S. Cataplexy is seen in an estimated
70% of narcolepsy patients and is a sudden reduction or loss of
muscle tone while a patient is awake, typically triggered by strong
emotions such as laughter, fear, anger, stress, or excitement.
Narcolepsy interferes with cognitive, psychological, and social
functioning, increases the risk of work- and driving-related
accidents, and is associated with a 1.5-fold higher mortality
rate.
About
AXS-12
AXS-12 (reboxetine) is a highly selective and
potent norepinephrine reuptake inhibitor under development for the
treatment of narcolepsy. AXS-12 modulates noradrenergic activity to
promote wakefulness, maintain muscle tone and enhance cognition.
AXS-12 has been granted U.S. Food and Drug Administration (FDA)
Breakthrough Therapy designation and Orphan Drug Designation for
the treatment of narcolepsy. AXS-12 is an investigational drug
product not approved by the FDA.
About Axsome
Therapeutics, Inc.
Axsome Therapeutics, Inc. is a biopharmaceutical
company developing novel therapies for the management of central
nervous system (CNS) disorders for which there are limited
treatment options. For the many people facing unsatisfactory
treatments for CNS disorders, Axsome accelerates the invention and
adoption of life-changing medicines. Axsome’s core CNS product
candidate portfolio includes five clinical-stage candidates,
AXS-05, AXS-07, AXS-09, AXS-12, and AXS-14. AXS-05 is being
developed for major depressive disorder (MDD), Alzheimer’s disease
(AD) agitation, and as a treatment for smoking cessation. AXS-07 is
being developed for the acute treatment of migraine. AXS-12 is
being developed for the treatment of narcolepsy. AXS-14 is being
developed for fibromyalgia. AXS-05, AXS-07, AXS-09, AXS-12, and
AXS-14 are investigational drug products not approved by the FDA.
For more information, please visit the Company’s website at
axsome.com. The Company may occasionally disseminate material,
nonpublic information on the company website.
Forward Looking Statements
Certain matters discussed in this press release
are “forward-looking statements”. We may, in some cases, use terms
such as “predicts,” “believes,” “potential,” “continue,”
“estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,”
“could,” “might,” “will,” “should” or other words that convey
uncertainty of future events or outcomes to identify these
forward-looking statements. In particular, the Company’s statements
regarding trends and potential future results are examples of such
forward-looking statements. The forward-looking statements include
risks and uncertainties, including, but not limited to, the
success, timing and cost of our ongoing clinical trials and
anticipated clinical trials for our current product candidates,
including statements regarding the timing of initiation, pace of
enrollment and completion of the trials (including our ability to
fully fund our disclosed clinical trials, which assumes no material
changes to our currently projected expenses), futility analyses and
receipt of interim results, which are not necessarily indicative of
the final results of our ongoing clinical trials, and the number or
type of studies or nature of results necessary to support the
filing of a new drug application (“NDA”) for any of our current
product candidates; our ability to fund additional clinical trials
to continue the advancement of our product candidates; the timing
of and our ability to obtain and maintain U.S. Food and Drug
Administration (“FDA”) or other regulatory authority approval of,
or other action with respect to, our product candidates (including,
but not limited to, with or without a special protocol assessment);
the potential for our clinical trials to provide a basis for
accelerated approval of our product candidates for the treatment of
several indications and accelerate their development timelines and
commercial paths to patients (including, but not limited to, with
or without a breakthrough therapy designation); the Company’s
ability to successfully defend its intellectual property or obtain
the necessary licenses at a cost acceptable to the Company, if at
all; the successful implementation of the Company’s research and
development programs and collaborations; the success of the
Company’s license agreements; the acceptance by the market of the
Company’s product candidates, if approved; the Company’s
anticipated capital requirements, including the Company’s
anticipated cash runway; unforeseen circumstances or other
disruptions to normal business operations arising from or related
to COVID-19; and other factors, including general economic
conditions and regulatory developments, not within the Company’s
control. The factors discussed herein could cause actual results
and developments to be materially different from those expressed in
or implied by such statements. The forward-looking statements are
made only as of the date of this press release and the Company
undertakes no obligation to publicly update such forward-looking
statements to reflect subsequent events or circumstance.
Axsome
Contact: Mark JacobsonChief Operating
OfficerAxsome Therapeutics, Inc.22 Cortlandt Street, 16th FloorNew
York, NY 10007Tel: 212-332-3243Email:
mjacobson@axsome.comwww.axsome.com
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